生产企业：BioMarin Pharmaceutical Inc.
BMN-351 is a fully modified 18-mer oligonucleotide with a TEG modification at its 5' end that binds to exon 51 of dystrophin pre-mRNA. It is under investigation for the treatment of Duchenne Muscular Dystrophy (DMD).
BMN-351 is a fully modified 18-mer oligonucleotide with a TEG modification at its 5' end that binds to exon 51 of dystrophin pre-mRNA. It is under investigation for the treatment of Duchenne Muscular Dystrophy (DMD).
共 1 项相关疾病
